Marisa Wexler, MS,  senior science writer—

The European Commission has approved Kalydeco (ivacaftor), by Vertex Pharmaceuticals, to treat infants as young as four months old with cystic fibrosis (CF) caused by certain mutations. The decision to extend Kalydeco’s label to younger babies, weighing at least 5 kg (about 11 lbs) and with relevant mutations, follows…

The National Organization for Rare Disorders (NORD)’s RareLaunch training program will host two days of free virtual workshops in December, with the aim of empowering leaders to start non-profit organizations and research programs to help people with rare diseases.  “The RareLaunch program is…

Oral lenabasum failed to significantly reduce the frequency of pulmonary exacerbations in people with cystic fibrosis (CF) treated in a Phase 2 trial, but new analyses suggest this failure could be attributed, at least in part, to differences among the patients that confounded results. The investigational therapy showed some…

A majority of rare disease patients using telehealth during the COVID-19 pandemic thought the experience positive, and many would like the option of continuing its use in future appointments, a series of surveys found. The surveys were conducted by the National Organization for Rare Disorders (NORD) and involved more than 800…

A Phase 2 clinical trial of oral lenabasum in people with cystic fibrosis (CF) failed to meet its primary goal of reducing pulmonary exacerbations, the investigational therapy’s developer, Corbus Pharmaceuticals, announced. “We are very disappointed that the study did not meet the primary endpoint,” Barbara White, MD, chief medical officer and…

Oral antibiotics are just as effective as intravenous (into-the-vein) antibiotics at killing Pseudomonas aeruginosa bacteria in people with cystic fibrosis (CF), a new study shows. This finding may allow people with CF to spend less time in the hospital to receive treatment, ultimately reducing treatment burden. The study,…

The U.S. Food and Drug Administration (FDA) has approved Vertex Pharmaceuticals‘ Kalydeco (ivacaftor) as a treatment for infants as young as four months with cystic fibrosis (CF) caused by certain mutations. The approval makes Kalydeco the first medication in its class to be approved for…

The European Medicines Agency (EMA) Committee for Medicinal Products for Human Use (CHMP) has recommended extending the licenses for Symkevi and Kalydeco, two cystic fibrosis (CF) treatments marketed by Vertex Pharmaceuticals, to allow these medications to be used in younger children. If approved by…

Adding pancreatic enzymes to “pre-digest” food was found to be an effective way to ensure nutrition in an infant with cystic fibrosis who was being fed through a feeding tube, according to a recent case report. The report, “Crushing pancreatic enzymes with enteral feeds in an extremely…

The European Medicines Agency (EMA) has validated an application that seeks to expand the approval of Kaftrio (ivacaftor/tezacaftor/elexacaftor) in combination with Kalydeco (ivacaftor) to treat cystic fibrosis (CF) in individuals 12 and older who have at least one copy of the F508del mutation…